Kraftsdorf

Researchers are evaluating various approved injectable and oral disease-modifying treatments (DMTs) in patients with relapsing multiple sclerosis (RMS) in Germany. This observational, non-interventional, multicenter, open-label study collects primary data prospectively over up to four years, alongside retrospective data. The study captures medical history, disease duration, laboratory values, disability scores (EDSS), MRI results, and relapse information to provide real-world insights into treatment use and outcomes. Patients receiving routine medical treatment with any approved injectable or selected oral DMTs—including ofatumumab, glatiramer acetate, interferon 21, teriflunomide, dimethyl fumarate, and diroximel fumarate—are enrolled without treatment allocation by the study. Two cohorts are observed: one treated primarily with injectable DMTs and another with injectable or oral DMTs. The core study period lasts about two years, with an optional extension providing an additional two years of observation, totaling up to four years. Follow-up visits and monitoring happen at the investigator's discretion and may include telemedicine. During the study, participants provide data through questionnaires and electronic case report forms. Routine clinical care procedures, such as diagnostic tests and monitoring, continue as usual. Researchers measure the proportion of patients continuing their baseline treatment at 24 months and collect ongoing clinical and imaging data. The study emphasizes real-world treatment patterns, safety, and disease activity over the extended follow-up period.

This research aims to evaluate the safety and effectiveness of a drug called DII235 in adults who have high levels of lipoprotein(a), a condition linked to lipoprotein disorder. The study focuses on adults aged 18 to 80 years who also have evidence of atherosclerotic cardiovascular disease or type 2 diabetes. This is a Phase 2 study designed to identify the best dose of DII235 and understand its impact on lipoprotein(a). Participants will be randomly assigned to receive either DII235 or a placebo in a controlled, double-blind manner to ensure unbiased results. The study involves administering DII235 or a saline placebo as solutions for injection. The trial is designed as a multi-center, randomized, double-blind, placebo-controlled, parallel-group, dose-finding study. Participants will receive different doses of DII235 or the placebo, and their responses will be compared over time to evaluate the drug's effects on lipoprotein(a) levels. The dosing and treatment schedules are carefully monitored to assess the safety, tolerability, and appropriate dosage levels of DII235. Participants will be followed and evaluated through various assessments, including measuring the percentage change from their baseline lipoprotein(a) levels between Day 60 and Day 180, and also between Day 60 and Day 360 for different doses. Safety and tolerability will be closely monitored throughout the study duration. The trial includes regular laboratory testing and clinical evaluations to track participant health and treatment response. Overall participation in the study spans several months to capture both short-term and longer-term effects of the treatment.

Researchers are evaluating the efficacy of claseprubart (DNTH103) compared to placebo in adults with chronic inflammatory demyelinating polyneuropathy (CIDP) in this Phase 3 study. The goal is to assess how well claseprubart works in treating this condition, which involves nerve inflammation leading to muscle weakness and sensory problems. The study consists of multiple periods: Part A is an open-label phase lasting up to 13 weeks where all participants receive claseprubart. Those who respond move to Part B, a randomized, double-blind, placebo-controlled phase lasting up to 52 weeks, where participants receive either claseprubart or placebo by infusion or injection. After Part B, eligible participants may join an optional open-label extension for up to 104 weeks. A safety follow-up period of 40 weeks follows the treatment phases. Participants will undergo various assessments including neurological evaluations and disease activity scoring. Researchers will monitor the time from the first dose to disease relapse as the main outcome. Additional safety and efficacy measures will be tracked throughout all study periods. Total participation may last over two years including extension and follow-up phases.

This research aims to evaluate the long-term safety and tolerability of pelacarsen (TQJ230) in adults with established cardiovascular disease and elevated Lipoprotein(a) who have completed the parent trial CTQJ230A12301. The study is an open-label extension following the phase 3 parent study, providing participants continued access to pelacarsen after the initial trial. Participants will receive pelacarsen 80 mg by subcutaneous injection once a month during this open-label extension. The study is single-arm and multicenter, focusing on continued treatment with pelacarsen for up to 36 months after completion of the parent study. Throughout the study, participants will be monitored regularly to assess safety and tolerability, with particular attention to adverse events occurring up to 36 months. Researchers will collect data on health status throughout this period to understand the long-term effects of pelacarsen in this patient population.

Researchers are evaluating the real-world effectiveness of efgartigimod in treating adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). The study aims to understand how participants experience treatment, describe their treatment journey, and assess their use of healthcare services. This is a noninterventional, prospective study where treatment decisions are made by participants and their physicians based on standard care practices. Participants who start efgartigimod treatment according to their country's approved label for CIDP will be followed. The study does not involve assigning treatments but observes the outcomes of those receiving efgartigimod. The treatment is given subcutaneously as per local guidelines, and participants are monitored over time. During the study, participants will be followed for up to 2 years from their first efgartigimod dose. Researchers will track changes in clinical scores such as aINCAT and I-RODS, grip strength, and quality of life using the EQ-5D questionnaire. This monitoring helps understand the treatment's impact over time and includes assessments of health status and function throughout the follow-up period.